Trial Outcomes & Findings for An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations (NCT NCT02934698)

NCT ID: NCT02934698

Last Updated: 2018-08-06

Results Overview

Absolute change in percent predicted in 1 second FEV1 from baseline through week 24

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 2 participants
• Primary outcome timeframe: 24 weeks
• Results posted on: 2018-08-06

Participant Flow

Participant milestones

Measure	Ivacaftor There is only one arm to this study. The two sisters with Cystic Fibrosis will both receive Ivacaftor for 6 months for their treatment. Ivacaftor: Subjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts.
Overall Study STARTED	2
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations

Baseline characteristics by cohort

Measure	Ivacaftor n=2 Participants There is only one arm to this study. The two sisters with Cystic Fibrosis will both receive Ivacaftor for 6 months for their treatment. Ivacaftor: Subjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts.
Age, Continuous	37 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	2 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants

PRIMARY outcome

Timeframe: 24 weeks

Absolute change in percent predicted in 1 second FEV1 from baseline through week 24

Outcome measures

Measure	Ivacaftor n=2 Participants There is only one arm to this study. The two sisters with Cystic Fibrosis will both receive Ivacaftor for 6 months for their treatment. Ivacaftor: Subjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts.
Forced Expiratory Volume	4 percentage of predicted Interval 3.0 to 5.0

SECONDARY outcome

Timeframe: 24 weeks

Achievement of mycobacterial culture conversion (negative culture)

Outcome measures

Measure	Ivacaftor n=2 Participants There is only one arm to this study. The two sisters with Cystic Fibrosis will both receive Ivacaftor for 6 months for their treatment. Ivacaftor: Subjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts.
Sputum Results	2 Participants

SECONDARY outcome

Timeframe: 24 Weeks

Testing efficacy through gathering absolute change in sweat chloride from baseline through week 24

Outcome measures

Measure	Ivacaftor n=2 Participants There is only one arm to this study. The two sisters with Cystic Fibrosis will both receive Ivacaftor for 6 months for their treatment. Ivacaftor: Subjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts.
Sweat Chloride	-5.7 mmol/L Interval -8.0 to -3.5

Adverse Events

Ivacaftor

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Ivacaftor n=2 participants at risk There is only one arm to this study. The two sisters with Cystic Fibrosis will both receive Ivacaftor for 6 months for their treatment. Ivacaftor: Subjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts.
Infections and infestations Sinus congestion	100.0% 2/2 • Number of events 4 • 24 weeks
Skin and subcutaneous tissue disorders Folliculitis	50.0% 1/2 • Number of events 1 • 24 weeks
Cardiac disorders palpitations	50.0% 1/2 • Number of events 1 • 24 weeks

Additional Information

Patrick Flume

MUSC

Phone: 843-792-3167

Email: flumepa@musc.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place